Effective induction therapy for anti-SRP associated myositis in childhood: A small case series and review of the literature.
Effective induction therapy for anti-SRP associated myositis in childhood: A small case series and review of the literature.
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DOI：
10.1186/s12969-017-0205-x
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发表时间：
2017-10-31
期刊：
影响因子：
--
通讯作者：
UK Juvenile Dermatomyositis Research Group (UK and Ireland)
中科院分区：
文献类型：
--
作者：
Binns EL;Moraitis E;Maillard S;Tansley S;McHugh N;Jacques TS;Wedderburn LR;Pilkington C;Yasin SA;Nistala K;UK Juvenile Dermatomyositis Research Group (UK and Ireland)
抗信号识别颗粒相关肌病是幼年特发性炎症性肌炎的一个临床和组织病理学独特亚组，在儿童中未被充分认识，对常规一线治疗无效。我们提出了三个病例，其中缓解成功诱导使用强化康复联合治疗。报告了3例新患者。3例患者均表现为严重的、快速发作的近端肌病，CK明显升高，但无皮疹。组织学检查显示破坏性肌病，其特征为散在萎缩和坏死纤维，很少或没有炎症浸润。所有3例患者均对环磷酰胺、IVIG和利妥昔单抗联合强化物理治疗和甲氨蝶呤作为维持剂的诱导治疗有反应。我们的患者恢复了接近正常的力量（MMT > 70/80），与当前文献相比，>50%的病例报告了严重的残余无力。对儿科抗SRP肌炎进行了文献检索，截止日期为2016年6月;使用以下术语的组合筛选PubMed：信号识别颗粒、自身抗体、抗体、肌炎、肌肉疾病、骨骼肌、儿童、儿科、青少年。排除了外语文章。本文支持抗SRP肌炎与其他JIIM不同的假设。这是与JDM的一个重要区别，如果出现严重无力而无皮疹或肌酶高度升高（CK > 10，000 U/l），应考虑到这一点。需要更大的国际合作和长期随访数据来为这一罕见的患者群体制定最有效的治疗策略。
Anti-Signal Recognition Particle associated myopathy is a clinically and histopathologically distinct subgroup of Juvenile Idiopathic Inflammatory Myositis, which is under-recognised in children and fails to respond to conventional first line therapies. We present three cases where remission was successfully induced using combination therapy with intensive rehabilitation. Three new patients are reported. All 3 cases presented with profound, rapid-onset, proximal myopathy and markedly raised CK, but no rash. Histology revealed a destructive myopathy characterized by scattered atrophic and necrotic fibres with little or no inflammatory infiltrate. All 3 patients responded to induction with cyclophosphamide, IVIG and rituximab, in conjunction with intensive physiotherapy and methotrexate as the maintenance agent. Our patients regained near-normal strength (MMT > 70/80), in contrast with the current literature where >50% of cases reported severe residual weakness. A literature search on paediatric anti-SRP myositis was performed to June 2016; PubMed was screened using a combination of the following terms: signal recognition particle, autoantibodies, antibodies, myositis, muscular diseases, skeletal muscle, childhood, paediatric, juvenile. Articles in a foreign language were excluded. Nine case studies were found. This paper supports the hypothesis that anti-SRP myositis is distinct from other JIIM. It is an important differential to JDM and should be considered where there is severe weakness without rash or if highly elevated muscle enzymes (CK > 10,000 U/l) are found. Early identification is essential to initiate aggressive medical and physical therapy. Greater international collaboration and long-term follow-up data is needed to establish the most effective treatment strategy for this rare group of patients.
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影响因子：
3.9
作者：
Tansley, Sarah L.;Betteridge, Zoe E.;McHugh, Neil J.
通讯作者：
McHugh, Neil J.
影响因子：
27.4
作者：
Hengstman, G. J. D.;ter Laak, H. J.;van Engelen, B. G. M.
通讯作者：
van Engelen, B. G. M.
影响因子：
--
作者：
RIDER, LG;MILLER, FW;PLOTZ, PH
通讯作者：
PLOTZ, PH
影响因子：
3.8
作者：
Suzuki, Shigeaki;Ohta, Masayasu;Nishino, Ichizo
通讯作者：
Nishino, Ichizo
大量肌炎自身抗体识别信号识别颗粒的肌炎患者的纵向病程。
影响因子：
4.7
作者：
Pinal-Fernandez, Iago;Parks, Cassie;Werner, Jessie L.;Albayda, Jemima;Paik, Julie J.;Danoff, Sonye K.;Casciola-Rosen, Livia;Christopher-Stine, Lisa;Mammen, Andrew L.
通讯作者：
Mammen, Andrew L.